The frequency of off-label use lacking scientific support indicates an overwhelming need for better information on safety, outcomes, and costs. However, given finite resources there is a need to prioritize research on off-label drug use so that future efforts can target those drugs where research is likely to be most valuable. This project will provide an evidence-based analysis to develop a prioritized list of drugs used in adult outpatient care where future research on off-label uses is expected to generate the greatest return on research efforts. Key initial aspects of the project include a review of current sources of information on off-label use and the recruitment of a set of specialized individuals to form a Technical Expert Panel (TEP) to best inform the prioritization process and provide final consensus on a specific prioritized list of drugs. We will then analyze the National Disease and Therapeutic Index (NDTI) and DRUGDEX data to examine the frequency and associated scientific support of off-label drug use, create a set of conceptual criteria that can be used to rank the potential importance of drugs in terms of their off-label use, and identify criteria that are measurable with available data. Finally, we will construct a cumulative priority score of current drugs via quantitative analysis of measurable criteria, create a final prioritized drug list based on each of the final criteria and consensus from the Technical Expert Panel, and use a secondary data source (from the federal Ambulatory Care Surveys) to provide additional insights into the use of top priority drugs. This secondary data will provide a means to assess limitations of the primary data source, use an alternative method for defining off-label use, and cover outpatient settings under-represented in NDTI. The project will serve as an important first step in prioritizing and focusing future research on the safety, outcomes, and costs of off-label prescribing.

Specific Aims

Review current sources of information on off-label drug use, including the existing literature.

Identify a set of key individuals to form a Technical Expert Panel (TEP) that will best inform the prioritization process and to provide final consensus on a specific prioritized list of drugs.

Employ a theoretical framework to help evaluate the need and directions of future research on off-label prescribing. Specifically, this framework will identify criteria for prioritizing drugs used off-label that might not otherwise be identified.

Analyze the best available data on the frequency and associated scientific support of off-label drug use by focusing on commonly used drugs whose off-label use presents potential concerns.

Create a set of conceptual criteria that can be used to rank the potential importance of drugs in terms of their off-label use and identify criteria that are measurable with available data.